Here is a picture of hard work. This is the paper work of a program with the best pre-clinical data ever in SMA. Thousands of pages outlining results, clinical trial design and safety for Dr. Kaspar’s Systemic Delivery of the AAV9/SMN Gene Therapy Program for SMA.
The best part…………it has officially been received by The Food and Drug Administration. They have 30 days to respond. We hope this will turn out to be the final chapter in what has been a horrific novel.
Thank you to everyone who has stood by us from the beginning. This is a quantifiable result from your support!